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Related Concept Videos

siRNA - Small Interfering RNAs02:30

siRNA - Small Interfering RNAs

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Small interfering RNAs, or siRNAs, are short regulatory RNA molecules that can silence genes post-transcriptionally, as well as the transcriptional level in some cases. siRNAs are important for protecting cells against viral infections and silencing transposable genetic elements.
In the cytoplasm, siRNA is processed from a double-stranded RNA, which comes from either endogenous DNA transcription or exogenous sources like a virus. This double-stranded RNA is then cleaved by the...
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RNA interference (RNAi) is a cellular mechanism that inhibits gene expression by suppressing its transcription or activating the RNA degradation process. The mechanism was discovered by Andrew Fire and Craig Mello in 1998 in plants. Today, it is observed in almost all eukaryotes, including protozoa, flies, nematodes, insects, parasites, and mammals. This precise cellular mechanism of gene silencing has been developed into a technique that provides an efficient way to identify and determine the...
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RNA interference (RNAi) is a process in which a small non-coding RNA molecule blocks the post-transcriptional expression of a gene by binding to its messenger RNA (mRNA) and preventing the protein from being translated.
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Related Experiment Video

Updated: Mar 29, 2026

Preparation of Neutrally-charged, pH-responsive Polymeric Nanoparticles for Cytosolic siRNA Delivery
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Preparation of Neutrally-charged, pH-responsive Polymeric Nanoparticles for Cytosolic siRNA Delivery

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Recent advances in siRNA delivery.

Can Sarisozen, Giuseppina Salzano, Vladimir P Torchilin

    Biomolecular Concepts
    |November 27, 2015
    PubMed
    Summary

    RNA interference (RNAi) uses small interfering RNAs (siRNAs) for gene silencing. Recent advances focus on overcoming delivery challenges for therapeutic applications in human diseases.

    Area of Science:

    • Biotechnology
    • Molecular Biology
    • Genetics

    Background:

    • RNA interference (RNAi) is a natural gene-silencing mechanism discovered in plants.
    • Small interfering RNAs (siRNAs) derived from RNAi have emerged as potent tools in biomedical research.
    • Therapeutic applications target diseases linked to altered gene expression.

    Purpose of the Study:

    • To provide an update on recent advancements in RNAi-based therapeutics.
    • To highlight novel synthetic platforms for intracellular siRNA delivery.
    • To address obstacles in translating siRNA therapies to clinical practice.

    Main Methods:

    • Review of recent scientific literature on RNAi therapeutics.
    • Analysis of clinical trial data and pre-clinical study outcomes.

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  • Exploration of novel synthetic delivery systems for siRNAs.

    • Main Results:

    • Significant progress has been made in RNAi-based therapeutic development.
    • Promising data from ongoing clinical trials have been reported.
    • Novel synthetic platforms show potential for improved intracellular siRNA delivery.

    Conclusions:

    • RNAi therapeutics hold great promise for treating various human diseases.
    • Overcoming delivery challenges is crucial for clinical translation.
    • Continued innovation in synthetic platforms will advance RNAi-based medicine.